Lila Rieber

16 karmaJoined


I work in gene therapy and can tell you that it has huge risks, which means that it's mainly considered for diseases that have no other treatment and would otherwise be fatal. For example, the latest gene therapy trial for X-linked myotubular myopathy has had four children die, probably because of AAV toxicity. The reason gene therapy is being considered for XLMTM is because it involves loss of function of a single gene in a specific cell type, as well as meeting the criteria I said before. Mood disorders aren't good candidates for gene therapy.